Pharming PHAR

Pharming Group N. V. is a global biotechnology company that develops and commercializes innovative therapies for rare and ultra-rare diseases with significant unmet medical need. The company focuses on immunological and genetic conditions where its scientific and commercial expertise can advance patient care over the long term. Pharming Group N. V. leverages its capabilities across clinical development manufacturing regulatory affairs and commercialization to deliver treatments for conditions such as hereditary angioedema and activated phosphoinositide 3-kinase delta syndrome.

Pharming Group N. V. generates revenue primarily through the sale of its two approved commercialized therapies RUCONEST® and Joenja®. RUCONEST® is a recombinant C1 esterase inhibitor protein replacement therapy indicated for the treatment of acute hereditary angioedema attacks in adult and adolescent patients. Joenja® is an oral selective phosphoinositide 3-kinase delta inhibitor approved as the first and only targeted treatment for activated phosphoinositide 3-kinase delta syndrome an ultra-rare primary immunodeficiency in adult and pediatric patients. The company commercializes these products through its own sales and marketing infrastructure in key markets including the United States and the United Kingdom.

The company operates through the following segments: commercial and pipeline.

• The commercial segment includes the marketing and sale of approved therapies RUCONEST® for hereditary angioedema and Joenja® for activated phosphoinositide 3-kinase delta syndrome supported by direct sales teams and commercial infrastructure in the United States and select international markets.
• The pipeline segment focuses on advancing clinical development programs including leniolisib for additional primary immunodeficiencies with immune dysregulation and napazimone KL1333 for mitochondrial DNA-driven primary mitochondrial disease through Phase II and pivotal clinical trials.

Pharming Group N. V. holds a differentiated position in the rare disease therapeutics industry as the developer of the first and only recombinant C1 esterase inhibitor for hereditary angioedema and the first and only approved targeted therapy for activated phosphoinositide 3-kinase delta syndrome. The company competes with established biopharmaceutical firms such as BioCryst BioMarin CSL Behring Intellia Ionis Kalvista Pharvaris and Takeda in the hereditary angioedema market while maintaining a unique therapeutic approach in activated phosphoinositide 3-kinase delta syndrome with no known active clinical development programs from competitors. Its competitive advantages stem from its focused rare disease strategy patient-centric commercial platform proprietary manufacturing technologies and high-value pipeline addressing significant unmet medical needs.

Pharming Group N. V. serves patients diagnosed with rare and ultra-rare diseases including those with hereditary angioedema and activated phosphoinositide 3-kinase delta syndrome. The company also supports caregivers healthcare providers and patient advocacy organizations through disease education diagnostic support programs and access initiatives. Specific customer groups include physicians specializing in immunology and hematology hospitals specialty clinics and national healthcare systems in markets where its therapies are approved and reimbursed.